An open-label study to collect safety and effectiveness information on long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy who have completed prior studies with vamorolone (The GUARDIAN Study)
Key facts
- Where
- Gent és Leuven, BelgiumSee the 2 sites
- Phase
- 4. fázis
Where in Belgium
- Universiteit GentGent, BelgiumNo longer taking part
- UZ LeuvenLeuven, BelgiumNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Santhera Pharmaceuticals (Schweiz) AG
- Trial code
- EU 2024-512828-12-00
- Record updated
- 2026. szeptember 21.
- Original description
To evaluate the safety of long-term treatment with vamorolone in boys with DMD regarding vertebral fracturesRead full original descriptionShow less
An open-label study to collect safety and effectiveness information on long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy who have completed prior studies with vamorolone (The GUARDIAN Study)
To evaluate the safety of long-term treatment with vamorolone in boys with DMD regarding vertebral fractures
Inclusion: Participant and/or participant's parent(s) or legal guardian has provided written informed consent; Participant has previously completed either the VBP15-LTE or VBP15-004 study, and transitioned through the CUP, NPP or EAP; Participant is on vamorolone on the day of enrolment; Participant and parent / legal guardian are willing and able to comply with the protocol schedule, assessments and requirements.
Exclusion: Vamorolone treatment discontinued for ≥6 months within the year prior to enrolment for a non-safety reason, or vamorolone treatment previously discontinued at any time for a safety reason; Any medical condition, which in the opinion of the Investigator, would affect study participation, performance or interpretation of study assessments; Severe hepatic impairment.